Pledge, this is very interesting, thanks for sharing. “by...

Pledge, this is very interesting, thanks for sharing.

“by getting pediatric approval, we will establish a new standard of care”

“[companies] will have to go head to head against our product to find a role in the pediatric form”

“there will not be competitors to our product in thismarket [adult third-line therapy]”

This sounds great for Mesoblast, at least for the next 12 months or so. But have a read back on what we have discussed previously regarding Cynata’s approach to aGvHD. As I have maintained, a label extension for Ryoncil to adult SR-aGvHD will probably impact Cynata’s Phase 2 trial recruitment rates in the US (8/29 sites worldwide). But the trial will complete and readout at some point, and (barring a miracle and going for approval straight away) the Phase 3 design will begin.

Recall that Cynata are treating patients classified as HIGH RISK at the point of developing aGvHD. This is at least 72 hours BEFORE they become classified as Steroid Resistant. Now that there is an established Standard of Care in pediatric patients, Cynata may look at the pediatric market, as a Phase 3 trial design can include Ryoncil as a standard of care for when the patient becomes SR, the same way the Phase 2 trial has Rux as a standard of care at that point in adults. They can do the same in adults (allowing the use of Rux at SR and Ryoncil as a 3^rd line therapy), that way there are no ethical issues with running the trial as a Randomised, controlled trial. The best kind of trial to truly understand how effective a treatment is.

Silviu may be right, it may be very difficult for another competitor to enter the market as the third-line therapy in adults with SR-aGvHD. However, that’s not the market Cynata is targeting. If the results (prob P2 and P3) show CYP-001 is effective, it doesn’t matter what patents Mesoblast have, Cynata’s therapy will enter the market at the point a patient is classified as High Risk. I think this move by Ross to pivot the company from targeting SR to HR has been extremely underrated because the share price has performed so poorly. But (if the treatment works), it may be worth hundreds of millions of dollars.